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Erythropoietic protoporphyria(EPP)

MedGen UID:
56455
Concept ID:
C0162568
Disease or Syndrome
Synonyms: EPP; Erythrohepatic protoporphyria
SNOMED CT: Protoporphyria (51022005); EPP - erythropoietic protoporphyria (51022005); Erythropoietic protoporphyria (51022005); Erythrohepatic protoporphyria (51022005); Magnus syndrome (51022005)
 
Monarch Initiative: MONDO:0001676
OMIM®: 177000
OMIM® Phenotypic series: PS177000

Definition

Erythropoietic protoporphyria-1 is an inborn error of porphyrin metabolism caused by decreased activity of the enzyme ferrochelatase, the terminal enzyme of the heme biosynthetic pathway, which catalyzes the insertion of iron into protoporphyrin to form heme. EPP is characterized clinically by photosensitivity to visible light commencing in childhood, and biochemically by elevated red cell protoporphyrin levels (Todd, 1994). Genetic Heterogeneity of Erythropoietic Protoporphyria Also see X-linked erythropoietic protoporphyria (XLEPP; 300752), caused by mutation in the ALAS2 gene (301300) on chromosome Xp11, and EPP2 (618015), caused by mutation in the CLPX gene (615611) on chromosome 15q22. [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVErythropoietic protoporphyria
Follow this link to review classifications for Erythropoietic protoporphyria in Orphanet.

Professional guidelines

PubMed

Sweeney P, Gimenez LE, Hernandez CC, Cone RD
Nat Rev Endocrinol 2023 Sep;19(9):507-519. Epub 2023 Jun 26 doi: 10.1038/s41574-023-00855-y. PMID: 37365323
Dickey AK, Naik H, Keel SB, Levy C, Beaven SW, Elmariah SB, Erwin AL, Goddu RJ, Hedstrom K, Leaf RK, Kazamel M, Mazepa M, Philpotts LL, Quigley J, Raef H, Rudnick SR, Saberi B, Thapar M, Ungar J, Wang B, Balwani M; Porphyrias Consortium of the Rare Diseases Clinical Research Network
J Am Acad Dermatol 2023 Dec;89(6):1227-1237. Epub 2022 Aug 27 doi: 10.1016/j.jaad.2022.08.036. PMID: 36041558Free PMC Article
Langendonk JG
Br J Dermatol 2015 Jun;172(6):1481-1482. doi: 10.1111/bjd.13769. PMID: 26036158

Recent clinical studies

Therapy

Balwani M, Bonkovsky HL, Levy C, Anderson KE, Bissell DM, Parker C, Takahashi F, Desnick RJ, Belongie K; Endeavor Investigators
N Engl J Med 2023 Apr 13;388(15):1376-1385. doi: 10.1056/NEJMoa2208754. PMID: 37043653
Heerfordt IM, Lerche CM, Wulf HC
Photodiagnosis Photodyn Ther 2022 Jun;38:102793. Epub 2022 Mar 2 doi: 10.1016/j.pdpdt.2022.102793. PMID: 35245673
Bissell DM, Anderson KE, Bonkovsky HL
N Engl J Med 2017 Aug 31;377(9):862-872. doi: 10.1056/NEJMra1608634. PMID: 28854095
Goetze S, Elsner P
J Dtsch Dermatol Ges 2015 Dec;13(12):1250-3. doi: 10.1111/ddg.12809. PMID: 26612794
Schulenburg-Brand D, Katugampola R, Anstey AV, Badminton MN
Dermatol Clin 2014 Jul;32(3):369-84, ix. Epub 2014 May 5 doi: 10.1016/j.det.2014.03.001. PMID: 24891059

Prognosis

Bissell DM, Anderson KE, Bonkovsky HL
N Engl J Med 2017 Aug 31;377(9):862-872. doi: 10.1056/NEJMra1608634. PMID: 28854095
Arora H, Chacon AH, Choudhary S, McLeod MP, Meshkov L, Nouri K, Izakovic J
Int J Dermatol 2014 Jul;53(7):798-802. Epub 2014 Mar 6 doi: 10.1111/ijd.12408. PMID: 24602044
Santoro FA, Lim HW
Semin Cutan Med Surg 2011 Dec;30(4):229-38. doi: 10.1016/j.sder.2011.07.007. PMID: 22123421
Lecha M, Puy H, Deybach JC
Orphanet J Rare Dis 2009 Sep 10;4:19. doi: 10.1186/1750-1172-4-19. PMID: 19744342Free PMC Article
Czarnecki DB
Arch Dermatol 1980 Mar;116(3):307-11. PMID: 7369748

Clinical prediction guides

Ventura P, Brancaleoni V, Di Pierro E, Graziadei G, Macrì A, Carmine Guida C, Nicolli A, Rossi MT, Granata F, Fiorentino V, Fustinoni S, Sala R, Pinton PC, Trevisan A, Marchini S, Cuoghi C, Marcacci M, Corradini E, Sorge F, Aurizi C, Savino MG, Cappellini MD, Pietrangelo A
Eur J Dermatol 2020 Oct 1;30(5):532-540. doi: 10.1684/ejd.2020.3880. PMID: 33021473
Phillips JD
Mol Genet Metab 2019 Nov;128(3):164-177. Epub 2019 Apr 22 doi: 10.1016/j.ymgme.2019.04.008. PMID: 31326287Free PMC Article
Mizawa M, Makino T, Nakano H, Sawamura D, Shimizu T
Acta Derm Venereol 2019 Jun 1;99(7):634-639. doi: 10.2340/00015555-3184. PMID: 30938825
Cox TM
J Inherit Metab Dis 1997 Jun;20(2):258-69. doi: 10.1023/a:1005317124985. PMID: 9211198
Cripps DJ
IARC Sci Publ 1986;(77):549-66. PMID: 3298037

Recent systematic reviews

Heerfordt IM, Lerche CM, Philipsen PA, Wulf HC
Photodiagnosis Photodyn Ther 2024 Jun;47:104211. Epub 2024 May 9 doi: 10.1016/j.pdpdt.2024.104211. PMID: 38734198
Heerfordt IM, Philipsen PA, Lerche CM, Wulf HC
Exp Dermatol 2023 Jul;32(7):1042-1047. Epub 2023 Apr 13 doi: 10.1111/exd.14809. PMID: 37052136
Heerfordt IM, Lerche CM, Philipsen PA, Wulf HC
Biomed Pharmacother 2023 Feb;158:114132. Epub 2022 Dec 14 doi: 10.1016/j.biopha.2022.114132. PMID: 36525819
Noyman Y, Edel Y, Snast I, Sherman S, Kaftory R, Lapidoth M, Mimouni D, Hodak E, Levi A
Photodermatol Photoimmunol Photomed 2021 Sep;37(5):374-379. Epub 2021 Feb 21 doi: 10.1111/phpp.12667. PMID: 33556208
Rutter KJ, Ashraf I, Cordingley L, Rhodes LE
Br J Dermatol 2020 May;182(5):1092-1102. Epub 2019 Sep 12 doi: 10.1111/bjd.18326. PMID: 31278744

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